The role of real-world trials and single-arm trials in the drug approval process

Looking forward to the future, the trends and challenges regarding the increasing utilization of real-world data and single-arm trials in the oncology drug approval pathway by European Medicines Agency (EMA) is very important, especially in the context of personalized medicine that will become the future.

Dr. Francesco Pignatti, Scientific advisor for Oncology in the Human Medicine Division of the European Medicines Agency, is the guest of this TJ Talks episode - led by dr. Luca Moscetti, Adjunct Professor in Oncology at the Residency Program in Oncology at the University of Modena Reggio Emilia - that addresses the relevant question for cancer patients about the EMA’s position on the use of real-world data in drug approvals.

It emerged during the thick TJ Talks that for big changes in the use of real-world data much will also depend on the uncertainty one is willing to accept in a specific situation. Single-arm trial, for example, comes with a lot of uncertainty, mostly about the time related endpoints like survival, then you have to you tend to accept them more where you have high unmet medical needs.

The goal of the scientific community is to focus the efforts on what really matters, trying to be more efficient, cleverer and to design quality criteria that are fit for purpose and that are proportionate to the objectives. Regulators, academics and the sponsors need to facilitate this type of research, maximising the opportunities that come from its data.

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